Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
NKT Therapeutics
ClinicalTrials.gov Identifier:
NCT01783691
First received: February 1, 2013
Last updated: September 19, 2014
Last verified: April 2014
  Purpose

The purpose of this study is to determine the safety, pharmacokinetics, and pharmacodynamics of NKTT120 in adult patients with stable sickle cell disease.


Condition Intervention Phase
Sickle Cell Disease
Drug: NKTT120
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Single Ascending Dose Phase 1 Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Biologic Activity of Intravenous NKTT120 in Adults With Stable Sickle Cell Disease

Resource links provided by NLM:


Further study details as provided by NKT Therapeutics:

Primary Outcome Measures:
  • Safety as assessed by (1) adverse events (2) laboratory values (3) vital signs and (4) physical exam. [ Time Frame: 180 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Pharmacokinetics assessed by plasma drug concentration levels [ Time Frame: 0; 15, 30, 60 minutes; 3,6, hours; 1,2,3,7,14,30,60,90 days ] [ Designated as safety issue: No ]
  • Pharmacodynamics as measured by assessments of peripheral blood lymphocyte subsets [ Time Frame: 0, 0.25, 1, 2, 7, 14, 30, 60, 90, 120 days ] [ Designated as safety issue: No ]

Other Outcome Measures:
  • Pain scores as assessed by daily electronic diary [ Time Frame: Daily for 120 days ] [ Designated as safety issue: No ]
  • Quality of Life as assessed by two separate Quality of Life instruments [ Time Frame: 0, 30, 60, 90, 120 days ] [ Designated as safety issue: No ]
  • Lung function as measured by pulmonary function tests [ Time Frame: 0; 30 minutes; 30, 60, 90, 120 days ] [ Designated as safety issue: No ]

Estimated Enrollment: 21
Study Start Date: February 2013
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NKTT120 Drug: NKTT120

  Eligibility

Ages Eligible for Study:   18 Years to 60 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age 18 to 50 years
  • Subject has a confirmed diagnosis of HbSS or HbSβ0thal
  • Subject has stable SCD defined as not having acute VOC, ACS, or other major SCD associated event during the month prior to enrollment

Exclusion Criteria:

  • Subject had an SCD-related VOC or ACS that required hospitalization or treatment in acute care outpatient setting in the month prior to enrollment
  • Subject requires a program of prescheduled regularly administered packed red blood cell (pRBC) transfusions or Subject received a pRBC transfusion in the month prior to enrollment
  • Subject has evidence of latent or active tuberculosis
  • Subject has a major concurrent illness or medical condition
  • Subject is pregnant or nursing
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01783691

Locations
United States, California
Children's Hospital & Research Center at Oakland
Oakland, California, United States, 94609
United States, Maryland
Johns Hopkins School of Medicine
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
United States, Missouri
Washington University School of Medicine
St. Louis, Missouri, United States, 63110
United States, North Carolina
UNC Sickle Cell Program
Chapel Hill, North Carolina, United States, 27514
United States, Wisconsin
Blood Center of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
NKT Therapeutics
Investigators
Study Director: Rosemary Mazanet, MD NKT Therapeutics
  More Information

No publications provided

Responsible Party: NKT Therapeutics
ClinicalTrials.gov Identifier: NCT01783691     History of Changes
Other Study ID Numbers: 120-SCD1
Study First Received: February 1, 2013
Last Updated: September 19, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by NKT Therapeutics:
Sickle cell disease

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies

ClinicalTrials.gov processed this record on October 23, 2014